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To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04134299
Recruitment Status : Recruiting
First Posted : October 22, 2019
Last Update Posted : October 22, 2019
Sponsor:
Information provided by (Responsible Party):
Axcella Health, Inc

Brief Summary:
This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks. The study also assesses the effects of this amino acid composition on the structure and function of the vascular system. Physiological effects on structure and function will be assessed by Magnetic Resonance Imaging (MRI) to assess blood flow in the brain and kidneys and the 6-Minute walk with pulse oximetry. Changes in blood biomarkers of inflammation will also be assessed.

Condition or disease Intervention/treatment Phase
To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease Anemia, Sickle Cell Sickle Cell Anemia Sickle Cell Disorders Sickle Cell Disease Dietary Supplement: AXA4010 Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: An Open-Label 12-Week Food Study to Assess the Safety, Tolerability, And Physiological Effects of An Amino Acid Food Product, AXA4010, In Subjects With Sickle Cell Disease.
Actual Study Start Date : September 25, 2019
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: AXA4010
AXA4010
Dietary Supplement: AXA4010
The study will initiate with approximately eight (8) adult subjects (≥18 y/o) with SCD (Cohort 1). Each subject will receive AXA4010 twice a day (BID) for 12 weeks. Safety and tolerability will be monitored, and study procedures will be performed. Analysis of safety, tolerability and laboratory assessments at week 12 would be performed. Based on the 12-week observations from at least three subjects in Cohort 1, subsequent Cohorts 2 and 3 in Part II may be initiated.




Primary Outcome Measures :
  1. Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ] [ Time Frame: Baseline to Week 12 ]
    Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)


Secondary Outcome Measures :
  1. Change in Lactate Dehydrogenase (LDH) [ Time Frame: Baseline to Week 12 ]
  2. Change Indirect Bilirubin [ Time Frame: Baseline to Week 12 ]
  3. Change Absolute Reticulocyte Count [ Time Frame: Baseline to Week 12 ]
  4. Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only) [ Time Frame: Baseline to Week 12 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Willing to participate in the study and provide written informed consent
  • Male and female adolescent (ages 12 to 17 inclusive) and adult (≥ 18 years of age) subjects with a prior medically documented diagnosis of sickle cell disease (SS or β0 thalassemia)
  • For Cohort 2 adult subjects only, a Screening cerebral blood flow/ velocity by transcranial doppler (TCD) should be >110 cm/sec
  • Subjects must show active hemolysis at the time of screening based on each of the following parameters:

    • Lactate dehydrogenase (LDH) ≥ 2x above upper limits of normal (ULN) of the age- and gender-appropriate ranges
    • Indirect bilirubin > 2x of the age- and gender-appropriate ULN
    • Absolute reticulocyte counts > 2x of the age- and gender-appropriate ULN
  • Able to ambulate (without assistance or walkers etc.) in a 6- minute walk test

Exclusion Criteria:

  • Ten (10) or more Vascular-Occlusive Crisis (VOC)s that required hospital or emergency room (ER) visits within the past 12 months
  • Any clinically significant changes abnormalities on the screening 12-lead ECG
  • Estimated glomerular filtration rate <60mL/min/1.73m2 appropriately corrected
  • Hemoglobin (Hb) ≤6.0 g/dL at Screening
  • Subjects on chronic transfusion regimens, or who received a transfusion within the last 2 months prior to screening
  • Experienced VOC or any other sickle crises (eg acute chest syndrome, splenic sequestration, dactylitis, stroke) within the last 2 months prior to Screening
  • Current or history of significant alcohol consumption
  • Other poorly controlled medical conditions as judged by the Investigator
  • Known sensitivity and/or history of clinically significant food intolerance/allergies to proteins (including whey, soy, casein, amino acids, etc.)
  • Unable or unwilling to adhere to contraception requirements
  • Any contraindications to an MRI scan for Cohort 2 only
  • Any other condition that, in the opinion of the Investigator, renders the subject at risk for compliance, compromises the well-being of the subject, or hinders study completion

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04134299


Contacts
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Contact: Margaret Koziel, MD 857-320-2200 regulatory@axcella.com
Contact: Manu Chakravarthy, MD 857-320-2200 regulatory@axcella.com

Locations
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United States, Florida
Foundation for Sickle Cell Disease Research Not yet recruiting
Hollywood, Florida, United States, 33021
Contact: Gershwin Blyden, MD,PhD,PA    954-240-8266    gblyden@fscdr.org   
Advanced Pharma CR, LLC Recruiting
Miami, Florida, United States, 33147
Contact: Kimberly Cruz, MD    305-220-2727 ext 864    kcruz@advancedpharmacr.com   
United States, Georgia
Primary Care research Recruiting
Atlanta, Georgia, United States, 30312
Contact: Courtney Shelton, MD    678-705-2355    shelton315md@gmail.com   
United States, New Jersey
Newark Beth Israel Medical Center Not yet recruiting
Newark, New Jersey, United States, 07112
Contact: Alice Cohen, MD    973-926-7231    Alice.Cohen@rwgbh.org   
Sponsors and Collaborators
Axcella Health, Inc
Investigators
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Principal Investigator: Kimberly Cruz, MD Advanced Pharma CR, LLC

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Responsible Party: Axcella Health, Inc
ClinicalTrials.gov Identifier: NCT04134299    
Other Study ID Numbers: AXA4010-001
First Posted: October 22, 2019    Key Record Dates
Last Update Posted: October 22, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Axcella Health, Inc:
Amino Acid
Food Study
Additional relevant MeSH terms:
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Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn